Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Patients Less Than 1 Year of Age

Phase 1/2 Completed 16 enrolled Kyowa Kirin Pharmaceutical Development Ltd

NaSingle-groupOpen-labelTreatment

Summary

A Phase 1/2, Open-label, Multicenter, Non-randomized Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Paediatric Patients from Birth to Less than 1 Year of Age with X-linked Hypophosphatemia (XLH)

Timeline

Start: 2020-02-26
Primary completion: 2023-10-04
Completion: 2024-03-06

Publications

Linglart A, Emma F, Bacchetta J, de la Cerda F, Hayes W, Cheung M, Cocca A, Hogler W, Padidela R, Henderson L, Tudor A, Okada H, Sun W, Quattrocchi E. Safety, tolerability, pharmacokinetics, and efficacy of burosumab in infants with X-linked hypophosphataemia: an open-label, multicentre, non-randomised study. Lancet Diabetes Endocrinol. 2026 Jun;14(6):475-484. doi: 10.1016/S2213-8587(26)00013-6. Epub 2026 Apr 24.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Burosumab	Monoclonal antibody	0.2 mg/kg	Subcutaneous
Subject	Burosumab	Monoclonal antibody	0.4 mg/kg	Subcutaneous
Subject	Burosumab	Monoclonal antibody	0.8 mg/kg	Subcutaneous
Subject	Burosumab	Monoclonal antibody	2 mg/kg	Subcutaneous

Study to Assess the Safety, Tolerability, Pharmacokinetics and Efficacy of Burosumab in Patients Less Than 1 Year of Age

Summary

Timeline

Publications

Drugs

Indications